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ZA201603714B - Delivery,use and therapeutic applications of the crispr-cas systems and compositions for genome editing - Google Patents

Delivery,use and therapeutic applications of the crispr-cas systems and compositions for genome editing

Info

Publication number
ZA201603714B
ZA201603714B ZA2016/03714A ZA201603714A ZA201603714B ZA 201603714 B ZA201603714 B ZA 201603714B ZA 2016/03714 A ZA2016/03714 A ZA 2016/03714A ZA 201603714 A ZA201603714 A ZA 201603714A ZA 201603714 B ZA201603714 B ZA 201603714B
Authority
ZA
South Africa
Prior art keywords
delivery
compositions
methods
crispr
systems
Prior art date
Application number
ZA2016/03714A
Inventor
Le Cong
David Benjamin Turitz Cox
Matthias Heidenreich
Randall Jeffrey Platt
Lukasz Swiech
Feng Zhang
Original Assignee
Broad Inst Inc
Massachusetts Inst Technology
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Broad Inst Inc, Massachusetts Inst Technology filed Critical Broad Inst Inc
Publication of ZA201603714B publication Critical patent/ZA201603714B/en

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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12N2320/10—Applications; Uses in screening processes
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Bioinformatics & Cheminformatics (AREA)
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Abstract

The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.
ZA2016/03714A 2013-12-12 2016-05-31 Delivery,use and therapeutic applications of the crispr-cas systems and compositions for genome editing ZA201603714B (en)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201361915192P 2013-12-12 2013-12-12
US201361915107P 2013-12-12 2013-12-12
US201361915148P 2013-12-12 2013-12-12
US201361915176P 2013-12-12 2013-12-12
US201361915153P 2013-12-12 2013-12-12
US201361915145P 2013-12-12 2013-12-12
US201361915215P 2013-12-12 2013-12-12
PCT/US2014/070127 WO2015089462A1 (en) 2013-12-12 2014-12-12 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing

Publications (1)

Publication Number Publication Date
ZA201603714B true ZA201603714B (en) 2024-09-25

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ZA2016/03714A ZA201603714B (en) 2013-12-12 2016-05-31 Delivery,use and therapeutic applications of the crispr-cas systems and compositions for genome editing

Country Status (15)

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US (2) US11407985B2 (en)
EP (2) EP3653229A1 (en)
JP (3) JP7103750B2 (en)
KR (1) KR20160089527A (en)
CN (3) CN111206032B (en)
AU (3) AU2014361781B2 (en)
BR (1) BR112016013201B1 (en)
CA (1) CA2932478A1 (en)
DK (1) DK3079725T3 (en)
ES (1) ES2765481T3 (en)
IL (3) IL316141A (en)
MX (2) MX374529B (en)
SG (1) SG10201804976YA (en)
WO (1) WO2015089462A1 (en)
ZA (1) ZA201603714B (en)

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